NCT03384420

A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome

A Phase I/II, Open Label, Single Dose Clinical Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD (Autologous cd34+ Cells Enriched With Blood Derived Mitochondria) in Pediatric Patients With Pearson Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Minovia Therapeutics Ltd. · Industry
• Sex: All
• Age: 3 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes. These diseases may be inherited from the mother (mitochondrial inheritance) or non-inherited. The latter are ultra-rare pediatric diseases caused by a mutation or deletion of mtDNA, which develop into a systemic multi organ disease and eventually death. MNV-BM-BLD is a therapeutic process for enrichment of patient's peripheral hematopoietic stem cells with normal and healthy mitochondria derived from donor blood cells. The process, called mitochondria augmentation therapy, aims to reduce the symptoms of mitochondrial diseases.

Conditions

• Mitochondrial Diseases
• Pearson Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	CD34+ cells enriched with MNV-BLD	Transplantation of autologous stem cell enriched with MNV-BLD (blood-derived mitochondria)

Timeline

• Start date: 2019-02-13
• Primary completion: 2021-03-09
• Completion: 2021-03-09
• First posted: 2017-12-27
• Last updated: 2021-08-31

Locations

1 site across 1 country: Israel

Regulatory

• FDA-regulated drug study